CARLSBAD, Calif., April 14 /PRNewswire-FirstCall/ -- Isis
Pharmaceuticals, Inc. (Nasdaq: ISIS) announced additional results from its
ongoing Phase 2 open-label extension (OLE) study of mipomersen in patients
with familial hypercholesterolemia (FH). In FH patients exposed to
mipomersen for as long as 16 months, the safety profile of the drug
continues to be encouraging. Reductions in LDL-cholesterol were also
maintained in patients who had responded to treatment in their initial
study and continued on treatment.

In this OLE study, patients with FH who were initially enrolled in one
of two Phase 2 efficacy studies of mipomersen were allowed to enter the OLE
study. All patients in the study were being treated with maximum-tolerated
lipid-lowering therapy before and during the study. Most patients received
200 mg/week of mipomersen in addition to maximum-tolerated lipid-lowering
therapy. The median duration of continuous exposure in these patients was
38 weeks. The maximum continuous exposure was 64 weeks.

All patients in the study had the opportunity to self-administer
mipomersen. Although the study was not designed to measure efficacy, the
activity of mipomersen was clearly demonstrated and appeared to be well
maintained with longer-term dosing.

With respect to safety, the drug was well tolerated in longer-term
treatment. There were no new types of adverse events observed and rates of
adverse events decreased during the OLE study from those in the initial
efficacy studies.
The following additional observations were made in these patients:

-- Mild injection site reactions remain the most common side effect

observation.

-- Injection site reactions did not worsen with longer-term dosing and

were not more prevalent in patients who self-administered mipomersen.

-- The incidence and severity of ALT elevations was less in the OLE study

than in the initial studies.

-- The maximal ALT seen in this study was 172 U/L.

-- No patient experienced an elevation of transaminase (ALT) associated

with increased bilirubin (Hy's Law). There were no bilirubins greater

than the upper limit of normal (ULN) observed in any subject.

-- Several patients who experienced increased ALTs either in the OLE

study or in the initial studies continued to receive mipomersen and

experienced decline in ALTs. No patient who experienced increased

ALTs greater than three times the ULN in their initial trial had

increased ALT values when they were retreated with mipomersen in the

OLE study.

"This open-label extension study suggests that mipomersen can be
administered with a satisfactory safety profile and continued LDL
reductions for prolonged periods of time in patients with familial
hypercholesterolemia being treated with maximal doses of statins and other
lipid lowering agents," said Evan Stein, M.D., Ph.D., Director, Metabolic
and Atherosclerosis Research Center, Cincinnati, Ohio.

Conference Call Information

At 8:30 a.m. Eastern Time Monday, April 14, Isis will conduct a live
webcast conference call to discuss mipomersen safety results. Interested
parties may access the webcast at http://www.isispharm.com or listen to the
call by dialing 877-856-1960. A webcast replay will be available for a
limited time.

ABOUT MIPOMERSEN

On January 7, 2008, Isis announced the licensing of mipomersen to
Genzyme as the preferred partner to continue development, commercialize and
market the drug. Isis will transition development responsibility for
mipomersen to Genzyme over the next year. In addition to the up-front
payment ($150 million for Isis stock at $30 per share and $175 million
mipomersen license fee), Isis also has the opportunity to receive from
Genzyme up to $825 million in development and regulatory milestone payments
plus up to $750 million in commercial milestone payments. Genzyme and Isis
will share mipomersen profits. The profit share begins with a 70/30
Genzyme/Isis split and increases linearly to 50/50 when revenue reaches $2
billion.

Mipomersen is a second-generation antisense drug that reduces the
production of apoB-100, a protein critical to the synthesis and transport
of "bad" cholesterol. Cholesterol can be carried in the bloodstream in a
variety of forms, with high-density lipoprotein, or HDL-cholesterol, being
the good form, and low-density lipoproteins, or LDL-cholesterol, and very
low-density lipoproteins, or VLDL-cholesterol, being bad forms directly
involved in heart disease. Collectively lowering LDL-cholesterol,
VLDL-cholesterol, and other bad forms of cholesterol are a key component in
the prevention and management of cardiovascular disease.

Currently in Phase 3 development, mipomersen has been shown in Phase 2
trials to reduce cholesterol and other atherogenic lipids more than 40
percent beyond reductions achieved with standard lipid-lowering drugs,
enabling more patients to achieve LDL-cholesterol targets. These trials
have shown that treatment with mipomersen is well-tolerated, and that
mipomersen has an attractive safety profile, and works equally well in the
presence and absence of other lipid-lowering therapies including statins. A
weekly injectable therapeutic, mipomersen is being developed primarily for
patients at high cardiovascular risk who are unable to achieve target
cholesterol levels with statins alone or who are intolerant of statins.

Mipomersen's initial indication will be for patients with FH. There are
approximately 1.5 million people in the United States and Europe with FH,
an inherited disorder that causes exceptionally high levels of
LDL-cholesterol. After appropriate clinical development, the next
indication pursued for mipomersen will be for other patients with high
cholesterol at high risk of cardiovascular events.

ABOUT FAMILIAL HYPERCHOLESTEROLEMIA (FH)

FH patients have high blood concentrations of LDL-cholesterol due to a
genetic disorder which prevents proper metabolism of LDL-cholesterol. These
patients experience a markedly increased risk of premature cardiovascular
diseases (CVD) and CVD-related death. FH can be present in two forms:
homozygous (hoFH), where the same defective gene is inherited from both
parents, or heterozygous (heFH), where the defective gene is inherited from
only one parent so that some function is preserved. The homozygous form is
a very rare condition estimated to affect approximately one in a million
people. Children with hoFH are at high risk for early coronary events and
sudden death as young as age one. HeFH is more common, with a prevalence of
approximately one in 500, and patients with heFH also experience elevated
LDL-cholesterol and are at high risk for early coronary events. For
undiagnosed or untreated heFH, the cumulative risk of a coronary heart
disease by age 60 years is 60-85 percent among men and 30-50 percent among
women, with a mean age of onset of approximately 47 years.

ABOUT ISIS PHARMACEUTICALS, INC.

Isis is exploiting its expertise in RNA to discover and develop novel
drugs for its product pipeline and for its partners. The Company has
successfully commercialized the world's first antisense drug and has 19
drugs in development. Isis' drug development programs are focused on
treating cardiovascular and metabolic diseases. Isis' partners are
developing antisense drugs invented by Isis to treat a wide variety of
diseases. Ibis Biosciences, Inc., Isis' majority-owned subsidiary, is
developing and commercializing the Ibis T5000(TM) Biosensor System, a
revolutionary system to identify infectious organisms. Isis is a joint
owner of Regulus Therapeutics LLC, a joint venture focused on the
discovery, development and commercialization of microRNA therapeutics. As
an innovator in RNA-based drug discovery and development, Isis is the owner
or exclusive licensee of over 1,500 issued patents worldwide. Additional
information about Isis is available at http://www.isispharm.com.

This press release includes forward-looking statements regarding the
development, activity, therapeutic potential and safety of mipomersen in
treating patients with high cholesterol. Any statement describing Isis'
goals, expectations, financial or other projections, intentions or beliefs
is a forward-looking statement and should be considered an at-risk
statement, including those statements that are described as Isis' goals.
Such statements are subject to certain risks and uncertainties,
particularly those inherent in the process of discovering, developing and
commercializing drugs that are safe and effective for use as human
therapeutics, and in the endeavor of building a business around such
products. Isis' forward-looking statements also involve assumptions that,
if they never materialize or prove correct, could cause its results to
differ materially from those expressed or implied by such forward-looking
statements. Although Isis' forward-looking statements reflect the good
faith judgment of its management, these statements are based only on facts
and factors currently known by Isis. As a result, you are cautioned not to
rely on these forward-looking statements. These and other risks concerning
Isis' programs are described in additional detail in Isis' annual report on
Form 10-K for the year ended December 31, 2007, which is on file with the
SEC. Copies of this and other documents are available from the Company.

In this press release, unless the context requires otherwise, "Isis,"
"Company," "we," "our," and "us" refers to Isis Pharmaceuticals and its
subsidiaries.

Isis Pharmaceuticals is a registered trademark of Isis Pharmaceuticals,
Inc. Ibis Biosciences and Ibis T5000 are trademarks of Ibis Biosciences,
Inc. Regulus Therapeutics is a trademark of Regulus Therapeutics LLC.

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